CGTLive’s Weekly Rewind – July 28, 2023

Welcome to CGTLive™’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. First in Human: PROCLAIM and upliFT-D Trials Bring Gene Therapy to Frontotemporal Dementia

With up to 20% of incidences of FTD driven by genetic mutations, multiple gene therapy companies have initiated clinical trials of AAV-mediated treatments for a patient population with little to no options.

2. Thomas Crawford, MD, on Confirming Long-Term Efficacy of Nusinersen for SMA

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed updated data from the NURTURE study.

3. BioCardia's CardiAMP Heart Failure Cell Therapy Faces Uncertain Future

A DSMB futility analysis found that the trial was unlikely to meet its primary composite endpoint and trial enrollment has been paused.

4. Fiona Freeman, PhD, on Benefits of miR-29b in Osteosarcoma Models

The assistant professor at University College Dublin discussed identifying miR-29b as a target for research in osteosarcoma.

5. Excision's CRISPR Therapy Fast Tracked for HIV

The phase 1/2 trial evaluating EBT-101 dosed its first patient in September 2022.